Promoting the use of CRISPR

To promote the use of CRISPR gene editing technology and expand the portfolio of knock out cell lines available to the scientific community as disease models and compound screening tools, Horizon Discovery is offering the opportunity to access free CRISPR reagents to generate knockouts:

Available to all academic researchers for knockouts in human cell lines, the programme offers up to 5 genes per laboratory.

To learn more about the programme we spoke to Chris Thorne, Team Leader, Reagents Group at Horizon Discovery.

AB: Can you tell me more about Horizon’s background in CRISPR gene editing?

Chris Thorne (CT): Horizon has a heritage in gene editing that goes back to the start of operations in 2007. Since that time we have used gene editing tools such as rAAV and more recently CRISPR to generate hundreds of isogenic cell line pairs in over a hundred cellular backgrounds. That experience has allowed us to become true gene editing experts.

The recent emergence of CRISPR has been particularly exciting for Horizon as it is enabling anyone to get started in modifying cell lines, to drive their translational genomics research. To support the research community Horizon has invested in accessing three separate suites of CRISPR-related intellectual property (from Harvard University, The Broad Institute, and ERS Genomics), to allow our partners to have confidence that they have the freedom to pursue their research goals.

AB: What does the CRISPR Knockout Generation programme offer academics?

CT: Primarily, academics receive very low cost access to CRISPR reagents and expertise. They only pay shipping costs, and the workload for the academics is also low as we do the design and cloning. This also means they have a lower risk environment to get started with CRISPR gene editing. If an academic is planning to knock out a gene in a cell line, or has been thinking about getting started with gene editing, this is the perfect way to do so.

The programme also gives academics access to gUIDEbook, our CRISPR guide design tool (developed jointly with Desktop Genetics). We will be further improving gUIDEbook as we learn more about which guides work better than others, through hundreds of successful cell line development programs. A public version of gUIDEbook will be available on Horizon's website shortly.

The programme comprises:

• The initiative is available to all academic researchers for knockouts in human cell lines, up to a maximum of 5 genes per laboratory• Horizon will design 5 RNA guides per each gene to knockout, using gUIDEbook™, our in silico guide design software• We provide the RNA guides in all-in-one plasmids that express Cas9 wild type• Horizon will ask in return feedback on which guide or guides worked and a license to the knock out cell lines generated• Thousands of genes are covered by the program

AB: Is the use of CRISPR limited in academia at present?

CT: There is a lot of excitement amongst the academic community around CRISPR, but there are also concerns. Is it too challenging, will my designs work, is it worth the cost? We feel that this is a great way to get the community not just talking about CRISPR, but also doing it, and we're prepared to invest to support the use and 'democratisation' of the technology.

AB: What will Horizon receive for offering this to researchers?

CT: First, this will help to build the community of those actively performing gene editing. Secondly it will further inform us on guide design so that we can continue to improve, and finally Horizon is looking to license back cell lines from researchers. Horizon is currently a repository of high-quality, genomically validated cell lines, and we have a history of working with the academic community for their generation. A significant number of our cell lines come from academic partners. By licensing cell lines back to us, we can ensure that quality controlled low passage cell line stocks are available (and at a discount of up to 95% for academic customers). The originating lab will receive a 5% royalty on every license taken by Horizon's customers for their cell line.

AB: Horizon has worked closely with leading institutions on rAAV gene editing through your ‘centres of excellence’ programme, can you tell me more about the programme and what it has achieved?

CT: The Centres of Excellence Programme has enabled researchers across the world to develop cells lines for the benefit of their research programmes. The collaborative nature of the programme, in which academic research labs are supported by the technical expertise of the Horizon team, has enabled the use of gene editing technologies (AAV) to be utilised in a broad range of research areas including oncology and cardiovascular disease. This partnership enables the researcher to develop novel reagents at minimal cost and entirely bespoke to their research aims, and also ultimately allows Horizon to expand its gene editing knowledge and cell line catalogue, as resulting cell lines are in-licenced from our collaborators. At present Horizon has over 50 centres across the world that are involved in the programme.

Through sharing the advances in gene editing represented by its GENESIS™ technology platform and derived X-MAN™ cell lines with the global academic research community, Horizon wishes to facilitate novel discoveries in the fields of translational genomics, personalised medicine, biological drug production and general life science research.

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